Acoramidis: A New Hope for ATTR-CM Patients in the EU

The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of acoramidis for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM) in the EU. This positive opinion is a significant milestone for patients suffering from this progressive and fatal disease, as it paves the way for a new treatment option with the potential to improve clinical outcomes and enhance quality of life.

ATTR-CM is a rare, inherited disease caused by mutations in the transthyretin (TTR) gene, leading to the accumulation of abnormal proteins in the heart. This accumulation causes progressive heart failure, with limited treatment options currently available. Acoramidis, a near-complete (≥90%) stabilizer of TTR, has shown promising results in clinical trials, demonstrating its ability to reduce cardiovascular death and cardiovascular-related hospitalization in ATTR-CM patients.

The Phase 3 ATTRibute-CM study, which served as the basis for the CHMP's positive opinion, enrolled 632 patients with diagnosed ATTR-CM and NYHA class I-III symptoms. The study found that treatment with acoramidis 800 mg twice daily improved the hierarchical primary composite endpoint of all-cause mortality, cardiovascular hospitalizations, change from baseline in NT-proBNP, and change from baseline in 6-minute walk distance at 30 months when compared with placebo. Remarkably, the overall survival and cardiovascular hospitalization rates approached that of age-matched populations without ATTR-cardiomyopathy.



Acoramidis' rapid onset of clinical benefits, demonstrated in as few as 3 months, sets it apart from other ATTR-CM treatments. In the Phase 3 ATTRibute-CM study, acoramidis showed a 42% reduction in composite all-cause mortality (ACM) and recurrent cardiovascular-related hospitalization (CVH) events relative to placebo at Month 30, with a 50% reduction in the cumulative frequency of CVH events. This early and sustained improvement in time to first event (CVH or ACM) starting at Month 3 highlights acoramidis' potential as a first-line therapy for ATTR-CM.



The expected patient population for acoramidis in the EU is approximately 15,000 patients with ATTR-CM, representing a significant market opportunity. The positive CHMP opinion, which is expected to lead to approval by the European Commission, will enable BridgeBio and Bayer to launch the drug in the EU in the first half of 2025. The drug's near-complete stabilization of transthyretin (TTR) and demonstrated clinical benefits, such as reduced cardiovascular death and hospitalization, are expected to drive market penetration. Additionally, the drug's oral administration and generally well-tolerated profile may further enhance its market acceptance.

In conclusion, the CHMP's positive opinion for acoramidis marks a significant step forward in the treatment of ATTR-CM. With its near-complete TTR stabilization and demonstrated clinical benefits, acoramidis has the potential to improve the lives of thousands of patients in the EU. As the drug progresses towards approval and launch, investors should closely monitor its market performance and potential impact on the ATTR-CM landscape.